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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06366464




Registration number
NCT06366464
Ethics application status
Date submitted
9/10/2023
Date registered
16/04/2024

Titles & IDs
Public title
A Study of Pitolisant in Patients with Prader-Willi Syndrome
Scientific title
A Phase 3, Randomized, Double-Blind, Placebo-controlled, Efficacy and Safety Study of Pitolisant Followed by an Open-Label Extension in Patients with Prader-Willi Syndrome
Secondary ID [1] 0 0
HBS-101-CL-312
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Prader-Willi Syndrome 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Mental Health 0 0 0 0
Learning disabilities
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Pitolisant tablet
Other interventions - Placebo tablet

Experimental: Double-Blind Treatment Period Pitolisant - Pitolisant tablets administered once daily in the morning upon wakening

Placebo comparator: Double-Blind Treatment Period Placebo - Matching placebo administered tablets once daily in the morning upon wakening

Other: Open-Label Extension Period Pitolisant - Pitolisant tablets administered once daily in the morning upon wakening


Treatment: Drugs: Pitolisant tablet
Pitolisant tablet

Other interventions: Placebo tablet
Placebo tablet
Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change in severity of EDS as measured by Patient-Reported Outcomes Measurement Information System Bank v1.0 - Sleep-Related Impairment (PROMIS-SRI) T-score
Timepoint [1] 0 0
Baseline and end of the Double Blind Treatment Period (Day 77)
Secondary outcome [1] 0 0
Change in severity of irritable and disruptive behaviors as measured by the Aberrant Behavior Checklist-Community, Second Edition (ABC-C) Irritability domain
Timepoint [1] 0 0
Baseline and end of the Double Blind Treatment Period (Day 77)
Secondary outcome [2] 0 0
Change in overall severity of EDS as measured by the Caregiver Global Impression of Severity for Excessive Daytime Sleepiness (CaGI-S for EDS)
Timepoint [2] 0 0
Baseline and end of the Double Blind Treatment Period (Day 77)
Secondary outcome [3] 0 0
Change in overall severity of EDS as measured by the Clinical Global Impression of Severity for Excessive Daytime Sleepiness (CGI-S for EDS)
Timepoint [3] 0 0
Baseline and end of the Double Blind Treatment Period (Day 77)
Secondary outcome [4] 0 0
Change in overall severity of irritable and disruptive behaviors as measured by the Caregiver Global Impression of Severity (CaGI-S) for Irritable and/or Disruptive Behaviors
Timepoint [4] 0 0
Baseline and end of the Double Blind Treatment Period (Day 77)
Secondary outcome [5] 0 0
Change in severity of hyperphagia as measured by the Hyperphagia Questionnaire for Clinical Trials (HQ-CT), in conjunction with the Food Safe Zone Questionnaire (FSZQ)
Timepoint [5] 0 0
Baseline and end of the Double Blind Treatment Period (Day 77)
Secondary outcome [6] 0 0
Change in severity of EDS as measured by the Epworth Sleepiness Scale for Children and Adolescents (ESS-CHAD [parent/caregiver version]) total score
Timepoint [6] 0 0
Baseline and end of the Double Blind Treatment Period (Day 77)
Secondary outcome [7] 0 0
Change in severity of other behavioral problems as measured by the Aberrant Behavior Checklist-Community, Second Edition (ABC-C) Hyperactivity/Noncompliance, Inappropriate Speech, Social Withdrawal, and Stereotypic Behavior Domains
Timepoint [7] 0 0
Baseline and end of the Double Blind Treatment Period (Day 77)
Secondary outcome [8] 0 0
Percentage of patients reporting TEAEs
Timepoint [8] 0 0
Baseline up to Day 441

Eligibility
Key inclusion criteria
* Genetically confirmed diagnosis of PWS
* Excessive daytime sleepiness
* Has a consistent parent/caregiver (preferably the same person throughout the study) who is willing and able to complete the required study assessments.
* In the opinion of the Investigator, the patient/parent(s)/caregiver(s)/legal guardian(s) are capable of understanding and complying with the requirements of the protocol and administration of oral study drug.
Minimum age
6 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Has a diagnosis of sleep apnea (OSA, CSA) that is not adequately controlled
* Has a diagnosis of hypersomnia due to another sleep/medical disorder
* Participation in an interventional research study involving another investigational medication, device, or behavioral treatment within 30 days or 5 half-lives (whichever is longer) of the investigational medication prior to Screening

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
28/05/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/07/2027
Actual
Sample size
Target
134
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD
Recruitment hospital [1] 0 0
Queensland Children's Hospital - Brisbane
Recruitment postcode(s) [1] 0 0
- Brisbane
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Delaware
Country [4] 0 0
United States of America
State/province [4] 0 0
Georgia
Country [5] 0 0
United States of America
State/province [5] 0 0
Illinois
Country [6] 0 0
United States of America
State/province [6] 0 0
Indiana
Country [7] 0 0
United States of America
State/province [7] 0 0
Maryland
Country [8] 0 0
United States of America
State/province [8] 0 0
Minnesota
Country [9] 0 0
United States of America
State/province [9] 0 0
New York
Country [10] 0 0
United States of America
State/province [10] 0 0
Ohio
Country [11] 0 0
United States of America
State/province [11] 0 0
Texas
Country [12] 0 0
United States of America
State/province [12] 0 0
Wisconsin

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Harmony Biosciences Management, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Ann Adee
Address 0 0
Country 0 0
Phone 0 0
773-383-6258
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT06366464